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Rare Diseases

Rare Disease Clinical Trial Expertise

An Opportunity for Real Impact

Creating a new therapy to treat a rare disease has the potential to make a significant impact on the lives of patients suffering from orphan conditions. The process to achieve this goal is substantially different and often much more challenging than what typically characterizes traditional drug development.

Despite the challenges, PPD is committed to advancing rare disease research and helping clients navigate the complexities associated with rare disease trials.

Rare Disease Framework

PPD deploys a four-part framework for rare disease studies that ensures the application of best practices in study development. The key elements of this framework include:

  1. Innovative and executable development plans – Deploying innovative, patient-centric development plans to navigate challenges and support clients in the development of clinical programs through:
    • Advancing patient pathways to understand diagnosing, protocols and treatments and possible engagement points with patients
    • Engaging key opinion leaders and patient advocacy groups to bring the patient and caregiver voice into trials design and execution
    • Using bayesian and adaptive design approaches when appropriate
    • Employing innovative approaches to data capture when needed to create positive experiences for patients and investigators
  2. Rigorous feasibility and study planning – identifying the right sites and finding and enrolling the patients crucial to a study’s success
    • Employing rigorous feasibility and study planning processes – identifying the right sites and finding and enrolling the right patients that are crucial to a study’s success
    • Mapping patient pathways for each indication to determine where and how to get patients into trials and what the trial experience will be like for the patient
    • Using data-driven modeling and feasibility to test study design and enrollment scenarios
    • Leveraging extensive experience to predict realistic enrollment rates and choose optimal locations for studies
    • Utilizing the rare disease and pediatrics center of excellence’s expertise in study planning
    • Employing high-touch site, investigator, lab and patient engagement services
    • Leveraging innovative data sources to drive site and patient identification
  3. Study execution – providing unmatched patient focus to retain and consistently engage patients and sites by:
    • Focusing on high-touch patient engagement and assurance of ongoing physician engagement
    • Utilizing patient retention approaches that ease patient burden including patient concierge services, such as travel support, home health care, reimbursement and one-on-one patient contact
    • Acknowledging the needs of sites that conduct rare disease studies by using site support approaches
    • Continuously engaging the rare disease and pediatrics center of excellence to proactively identify and mitigate challenges
    • Focusing on the patient experience to keep patients enrolled
    • Conducting standardized site training to ensure quality execution, data quality and compliance
    • Consistently engaging with sites for rapid issue escalation
    • Accessing real-time study data via preclarus®
  4. Proactive post-study planning – leveraging key partnerships to plan for the future by:
    • Focusing on longitudinal site relationships for entire programs, not single trials, which allows for:
      • Planning the entire landscape of a program and all needs of particular patients that may enroll in natural history studies or patient registries
      • Partnering with clients at the beginning of a program to plan and optimize patient flow through the entire program
      • Supporting investigational product access programs for patients after trial completion
      • Engaging with patients and investigators post-study to create ongoing network of advocates
      • Conducting post-market safety analyses
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